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Momelotinib in Transfusion-Dependent Adults With Primary Myelofibrosis (PMF) or Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis (Post-PV/ET MF)

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02515630
First Posted: August 5, 2015
Last Update Posted: August 23, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Gilead Sciences
  Purpose
This study will evaluate the transfusion independence response rate in transfusion-dependent adults with myelofibrosis after treatment with momelotinib (MMB).

Condition Intervention Phase
Primary Myelofibrosis (PMF) Post-polycythemia Vera (Post-PV) Myelofibrosis Postessential Thrombocythemia (Post-ET) Myelofibrosis Drug: MMB Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2, Open-label, Translational Biology Study of Momelotinib in Transfusion-Dependent Subjects With Primary Myelofibrosis (PMF) or Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis (Post-PV/ET MF)

Resource links provided by NLM:


Further study details as provided by Gilead Sciences:

Primary Outcome Measures:
  • Transfusion independence response rate by Week 24 [ Time Frame: Up to 24 weeks ]
    Transfusion independence response rate is defined as becoming transfusion independent for ≥ 12 weeks at any time on study.


Secondary Outcome Measures:
  • Transfusion response rate by Week 24 [ Time Frame: Up to 24 weeks ]
    Transfusion response rate is defined as becoming not transfusion dependent for ≥ 8 weeks at any time on study

  • Baseline and change in marker of anemia [ Time Frame: Up to 24 weeks ]
  • Change in pharmacodynamics biomarker [ Time Frame: Up to 24 weeks ]
  • Splenic response rate at Week 24 [ Time Frame: Week 24 ]
    Splenic response rate is defined as ≥ 35% reduction in spleen volume from baseline as measured by MRI.

  • Response rate in total symptom score (TSS) at Week 24 [ Time Frame: Week 24 ]
    Response rate in TSS is defined as achieving a ≥ 50% reduction from baseline in TSS as measured by the modified Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPNSAF TSS) diary.

  • Maximum observed plasma concentration (Cmax) of MMB [ Time Frame: Predose and 2, 4, 6, hours postdose ]
  • Last observable concentration (Clast) of MMB [ Time Frame: Predose and 2, 4, 6, hours postdose ]
  • Observed drug concentration at the end of the dosing interval (Ctau) for MMB [ Time Frame: Predose and 2, 4, 6, hours postdose ]
  • Area under the concentration-time curve from time zero to time of the last quantifiable concentration (AUClast) for MMB [ Time Frame: Predose and 2, 4, 6, hours postdose ]
    AUClast is defined as the concentration of drug from time zero to the last quantifiable concentration.

  • Change in circulating cytokine and inflammatory markers [ Time Frame: Up to 24 weeks ]

Enrollment: 41
Actual Study Start Date: January 29, 2016
Study Completion Date: August 15, 2017
Primary Completion Date: July 18, 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Momelotinib
MMB for 24 weeks (± 7 days)
Drug: MMB
Momelotinib (MMB) tablet administered orally once daily
Other Names:
  • GS-0387
  • CYT387

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Diagnosis of PMF or Post PV/ET-MF
  • Requires myelofibrosis therapy, in the opinion of the investigator
  • High risk OR intermediate-2 risk defined by dynamic international prognostic scoring system (DIPSS) OR intermediate-1 risk defined by DIPSS and associated with symptomatic splenomegaly and/or hepatomegaly
  • Transfusion dependent at baseline, defined as ≥ 4 U red blood cell (RBC) transfusion in the 8 weeks prior to first dose of MMB
  • Acceptable organ function as evidenced by the following:

    • Platelet Count ≥ 50 x 10^9/L
    • Aspartate aminotransferase (AST/SGOT) and alanine aminotransferase (ALT/SGPT) ≤ 3 x upper limit of normal (ULN) or AST or ALT ≤ 5 x ULN if liver is involved by disease process as judged by the investigator
    • Serum creatinine ≤ 2.0 mg/dL or calculated creatinine clearance of ≥ 60 mL/min
    • Direct bilirubin ≤ 2.0 x ULN
  • Life expectancy of > 24 weeks
  • Males and females of childbearing potential who engage in heterosexual intercourse must agree to use protocol specified method(s) of contraception
  • Lactating females must agree to discontinue nursing before MMB administration
  • Able to understand and willing to sign the informed consent form

Key Exclusion Criteria:

  • Prior splenectomy
  • Splenic irradiation within 3 months prior to the first dose of MMB
  • Prior treatment with MMB
  • Known positive status of human immunodeficiency virus (HIV)
  • Chronic active or acute viral hepatitis A, B, or C infection (testing required for hepatitis B and C), or hepatitis B or C carrier
  • Use of strong cytochrome P450 3A4 (CYP3A4) inducer within 2 weeks prior to the first dose of MMB
  • Uncontrolled intercurrent illness per protocol
  • Treatment with a Janus kinase (JAK) inhibitor within 21 days of the planned first dose of MMB
  • Presence of peripheral neuropathy ≥ Common Terminology Criteria for Adverse Events (CTCAE) Grade 2
  • Unwilling or unable to undergo a MRI per requirements in the study protocol
  • Unwilling to consent to genomics sampling

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02515630


Locations
United States, Arizona
Phoenix, Arizona, United States
United States, California
Los Angeles, California, United States
Orange, California, United States
United States, Florida
Jacksonville, Florida, United States
United States, Maryland
Baltimore, Maryland, United States
United States, Michigan
Ann Arbor, Michigan, United States
United States, Missouri
Saint Louis, Missouri, United States
United States, New York
New York, New York, United States
The Bronx, New York, United States
United States, North Carolina
Durham, North Carolina, United States
United States, Ohio
Cleveland, Ohio, United States
United States, Texas
Houston, Texas, United States
Canada, Ontario
Toronto, Ontario, Canada
Sponsors and Collaborators
Gilead Sciences
Investigators
Study Director: Gilead Study Director Gilead Sciences
  More Information

Responsible Party: Gilead Sciences
ClinicalTrials.gov Identifier: NCT02515630     History of Changes
Other Study ID Numbers: GS-US-352-1672
First Submitted: August 3, 2015
First Posted: August 5, 2015
Last Update Posted: August 23, 2017
Last Verified: August 2017

Additional relevant MeSH terms:
Primary Myelofibrosis
Polycythemia
Polycythemia Vera
Thrombocytosis
Thrombocythemia, Essential
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases
Bone Marrow Neoplasms
Hematologic Neoplasms
Neoplasms by Site
Neoplasms
Blood Platelet Disorders
Blood Coagulation Disorders
Hemorrhagic Disorders